Interim results from a Phase 2 study in C9orf72-related ALS/FTD patients (NCT04993755) are highly promising, suggesting that TPN-101 may favorably impact biomarkers of neurodegeneration and inflammation, such as neurofilament light (NfL), neurofilament heavy chain (NfH), and interleukin-6 (IL-6). The gene discussed is NEFL; the disease is amyotrophic lateral sclerosis.